Abeona Therapeutics and Beacon Therapeutics Announce Non-Exclusive Agreement for Beacon to Evaluate Therapeutic Potential of Abeona's Patented AAV204 Capsid for Select Ophthalmology Indications

Core Viewpoint - Abeona Therapeutics and Beacon Therapeutics have entered into an agreement for the evaluation of Abeona's AAV204 capsid for potential gene therapies targeting ophthalmic diseases, highlighting the collaboration's focus on addressing high unmet medical needs in this area [1][2]. Group 1: Agreement Details - Beacon will evaluate the AAV204 capsid for a 12-month period for specific ophthalmology indications, with an option for a worldwide, non-exclusive license for up to five gene or disease targets [2]. - Abeona will receive an upfront payment upon Beacon's exercise of its licensing option, along with additional milestone payments and tiered royalties on worldwide net sales for licensed products incorporating AAV204 [2]. Group 2: AAV204 Capsid Characteristics - AAV204, part of the AIM™ capsid library licensed from the University of North Carolina, has demonstrated high transduction levels in the macula and optic nerve after pararetinal administration, and effective transduction in both inner and outer retina after intravitreal administration in preclinical studies [2][3]. - The AIM™ capsid library aims to improve delivery of genetic payloads to key tissues involved in genetic diseases, potentially evading immune responses associated with naturally-occurring AAV vectors [3]. Group 3: Company Backgrounds - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including a candidate for recessive dystrophic epidermolysis bullosa and AAV-based therapies for ophthalmic diseases [4]. - Beacon Therapeutics, founded in 2023, is dedicated to developing gene therapies for retinal diseases, with existing programs targeting X-linked retinitis pigmentosa, dry age-related macular degeneration, and cone-rod dystrophy [5].